Hunter Syndrome News and Research

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Hunter syndrome, or mucopolysaccharidosis Type II, is a lysosomal storage disease caused by a deficient (or absent) enzyme, iduronate-2-sulfatase (I2S).
The syndrome is named after physician Charles A. Hunter (1873-1955), who first described it in 1917. Born in Scotland, Hunter emigrated to Canada and had a medical practice in Winnipeg, Manitoba.

Study offers a new way to treat children with bow hunter syndrome

Fusing the neck's top two vertebrae can prevent repeat strokes in children with bow hunter syndrome, a rare condition that affects a handful of U.S. pediatric patients each year, UT Southwestern researchers suggest in a recent study.

11 Feb 2021

Umbilical cord blood safely and effectively treats children with rare genetic disorders

Researchers at UPMC Children's Hospital of Pittsburgh found that infusing umbilical cord blood - a readily available source of stem cells - safely and effectively treated 44 children born with various non-cancerous genetic disorders, including sickle cell, thalassemia, Hunter syndrome, Krabbe disease, metachromatic leukodystrophy (MLD) and an array of immune deficiencies.

14 Jul 2020

Gene editing within a living person for the first time

Genetic editing has been performed in the laboratory until now. Living cells within Petri-dishes have been put under the microscope while gene editing tools such as CRISPR were used to cut, nick and replace parts of the DNA or genome that is defective with bits of DNA that is correct in its sequence. Now doctors in California have attempted to edit cells within a living person.

19 Nov 2017

Shire launches awareness campaign to improve lives of people with rare MPS

Shire plc announces a campaign to enable people to connect and share information about MPS (mucopolysaccharide) diseases around the world.

11 May 2016

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration has cleared the Company's Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I).

8 Feb 2016

Synageva announces submission of Kanuma application to Cofepris for treatment of LAL Deficiency

Synageva BioPharma Corp., a biopharmaceutical company developing therapeutic products for rare disorders, announced today the submission to the Comisión Federal para la Protección contra Riesgos Sanitarios in Mexico for Kanuma as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency), a rare genetic disease with significant morbidity and early mortality.

30 Mar 2015

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

ArmaGen, Inc., a privately held biotechnology company focused on developing novel therapies to treat severe neurological disorders, announced today that the Investigational New Drug (IND) application for the company's lead product candidate, AGT-182 for the treatment of Hunter syndrome, has been accepted by the U.S. Food and Drug Administration and is now active.

11 Dec 2014

Shire, ArmaGen partner to develop AGT-182 drug for treatment of Hunter syndrome

Shire plc, the global specialty biopharmaceutical company, and ArmaGen, a US privately held biotechnology company, today announced a worldwide licensing and collaboration agreement for AGT-182, an investigational enzyme replacement therapy (ERT) for the potential treatment of both the central nervous system (CNS) and somatic manifestations in patients with Hunter syndrome (MPS II).

23 Jul 2014

FDA grants orphan drug designation to ARMAGEN's AGT-182 for treatment of Hunter syndrome

ARMAGEN announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II).

19 Jul 2013

Renaissance in drug development for rare diseases

Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug manufacturers, who are ramping up research efforts and marketing new medicines that promise fuller lives for children and other patients with these heartbreaking conditions.

14 May 2013

GSK, Angiochem to develop and commercialize LSD treatments

Angiochem announced today a global collaboration with GlaxoSmithKline (GSK) to discover, develop and commercialize treatments for lysosomal storage diseases.

27 Feb 2012

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis."

19 May 2010

Genetic Disorder Tracker introduced by Guidepoint Global

Guidepoint Global, LLC, a leading primary research firm, today announced the launch of their Genetic Disorder Tracker, a monthly longitudinal data release that captures commercial trends, on a worldwide basis, associated with the treatment of eight rare genetic disorders: Gaucher's disease, Fabry's disease, PKU, MPS I (Hurler Syndrome), MPS II (Hunter Syndrome), MPS VI (Maroteaux-Lamy Syndrome), Niemann-Pick disease and Pompe disease.

25 Feb 2010